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Abstract：
Ribonucleic acid interference (RNAi) is an innovative treatment strategy for a myriad of indications. Non-viral synthetic nanoparticles (NPs) have drawn extensive attention as vectors for RNAi due to their potential advantages, including improved safety, high delivery efficiency and economic feasibility. However, the complex natural process of RNAi and the susceptible nature of oligonucleotides render the NPs subject to particular design principles and requirements for practical fabrication. Here, we summarize the requirements and obstacles for fabricating non-viral nano-vectors for efficient RNAi. To address the delivery challenges, we discuss practical guidelines for materials selection and NP synthesis in order to maximize RNA encapsulation efficiency and protection against degradation, and to facilitate the cytosolic release of oligonucleotides. The current status of clinical translation of RNAi-based therapies and further perspectives for reducing the potential side effects are also reviewed.
摘要：
核糖核酸干扰（RNAi）是一种针对多种适应症的创新治疗策略。非病毒合成纳米粒子(NPs)作为RNAi的载体因其潜在的优势而受到广泛关注。包括提高安全性，输送效率高，经济可行性强。然而,RNAi的复杂自然过程和寡核苷酸的易感性质使得NP服从于特定的设计原则和实际制造的要求。这里,我们总结了制造有效RNAi的非病毒纳米载体的要求和障碍。为了应对交付挑战，我们讨论了材料选择和NP合成的实用指南，以最大限度地提高RNA封装效率和防止降解，并促进寡核苷酸的胞浆释放。还回顾了基于RNAi的疗法的临床翻译的现状以及减少潜在副作用的进一步观点。
